Table 1. Baseline characteristics of included studies.
| RCT name | Patient, n | FH subtype | Age, years | Male, % | White, % | HTN, % | DM, % | LDL, mg/dL | TG, mg/dL | Lp(a), mgdL*; nmol/l† | ApoB, mg/dL | HIS use, % | EZ use, % |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Type of intervention | |||||||||||||
| ODYSSEY ESCAPE 24 | 62 | HeFH | 58.7 | 58.0 | 96.8 | NA | NA | 181.11 | 196.87 | 32.76* | 143.4 | 40.3 | NA |
| Alirocumab (150 mg Q2W) | |||||||||||||
| ODYSSEY FH I 16 | 486 | HeFH | 52.0 | 56.38 | 91.3 | 43.2 | 11.7 | 144.6 | 127.83 | 49.96* | 114.3 | 83.54 | 57.2 |
| Alirocumab (75 mg Q2W) | |||||||||||||
|
ODYSSEY FH II
16 |
249 | HeFH | 53.2 | 52.61 | 98.0 | 32.5 | 4.0 | 134.4 | 121.23 | 50.23* | 107.9 | 88.35 | 66.26 |
| Alirocumab (75 mg Q2W) | |||||||||||||
| ODYSSEY HIGH FH 25 | 107 | HeFH | 50.55 | 53.27 | 87.85 | 57.0 | 14.0 | 197.85 | 129.83 | 27.0* | 140.92 | 72.9 | 24.3 |
| Alirocumab (150 mg Q2W) | |||||||||||||
| ODYSSEY HoFH 26 | 69 | HoFH | 43.38 | 49.27 | 78.26 | NA | NA | 282.7 | 107.17 | 34.8* | 186.93 | 85.5 | 72.46 |
| Alirocumab (150 mg Q2W) | |||||||||||||
| Santos 2024 22 | 153 | HeFH | 12.90 | 43.14 | 81.7 | NA | NA | 174.23 | 79.18 | 22.65* | 117.41 | 15.03 | 13.72 |
| Alirocumab (40 mg Q2W; 75 mg Q2W; 150 mg Q4W; 300 mg Q4W) | |||||||||||||
| Stein 2012 #1 23 | 77 | HeFH | 53.4 | 61.0 | 95.0 | NA | 4.0 | 155.07 | 119.57 | NA | 127.0 | 77.0 | 71.0 |
| Alirocumab (150 mg Q4W; 200 mg Q4W; 300 mg Q4W; 150 mg Q2W) | |||||||||||||
| Stein 2012 #2 14 | 21 | HeFH | 40.43 | 80.86 | 85.71 | NA | NA | 133.56 | NA | NA | 112.0 | 52.38 | NA |
| Alirocumab (50 mg; 100 mg; 150 mg)‡ | |||||||||||||
| Ridker 201820 (SPIRE trials) | 1578 | HeFH | 57.60 | 58.11 | NA | 59.25 | 24.9 | 151.36 | 139.04 | 40.41* | 116.46 | 80.9 | 46.94 |
| Bococizumab (150 mg Q2W) | |||||||||||||
| HAUSER-RCT 21 | 157 | HeFH | 13.7 | 43.7 | 85.0 | 3.35 | 0.66 | 184.32 | 86.26 | 44.9† | NA | 16.6 | 13.4 |
| Evolocumab (420 mg Q4W) | |||||||||||||
| RUTHERFORD 27 | 167 | HeFH | 49.58 | 53.29 | 88.62 | NA | NA | 157.39 | 124.0 | 69.83† | 123.0 | 89.82 | 64.67 |
| Evolocumab (350 mg Q4W; 420 mg Q4W) | |||||||||||||
| RUTHERFORD-2 28 | 329 | HeFH | 51.15 | 57.75 | NA | NA | NA | 155.84 | 116.91 | 93.61† | 113.0 | 87.0 | 62.0 |
| Evolocumab (140 mg Q2W; 420 mg Q4W) | |||||||||||||
| TESLA 29 | 49 | HoFH | 31.0 | 51.22 | 90.0 | NA | NA | 348.03 | 106.28 | 92.67† | 210.0 | 94.0 | 92.0 |
| Evolocumab (420 mg Q4W) | |||||||||||||
| CREDIT-2 30 | 148 | HeFH | 49.41 | 52 | NA | NA | NA | 164.35 | 138.17 | 28.0* | 121.0 | 12.16 | 28.38 |
| Tafolecimab (150 mg Q2W; 450 mg Q4W) | |||||||||||||
| ORION-5 31 | 56 | HoFH | 42.7 | 39.3 | NA | 37.5 | 5.4 | 315.3 | NA | 85.0† | 203.1 | 100.0 | 66.1 |
| Inclisiran sodium (300 mg)§ | |||||||||||||
| ORION-9 32 | 482 | HeFH | 5m6.0 | 47.1 | 93.98 | 42.1 | 9.96 | 153.04 | 123.16 | 86.33† | 124.15 | 73.86 | 52.9 |
| Inclisiran sodium (300 mg)¶ | |||||||||||||
| ORION-15 33 | 56 | HeFH | †† | †† | †† | †† | †† | †† | †† | †† | †† | †† | †† |
| Inclisiran sodium (100 mg, 200 mg, 300 mg)** | |||||||||||||
| ORION-18 34 | 36 | HeFH | †† | †† | †† | †† | †† | †† | †† | †† | †† | †† | †† |
| Inclisiran sodium (300 mg)§§ |
Values are mean or %.
Measured in mg/dL
Measured in nmol/L.
Administered on days 1, 29 and 43.
Administered on days 1 and 90.
Administered on days 1, 90, 270 and 450.
Administered on days 1, 90, 270.
Baseline characteristics of FH subgroup in ORION-15 and ORION-18 are not reported, only for the overall population.
ApoB, apolipoprotein B; DM, diabetes mellitus; EZ, Ezetimibe; FH, familial hypercholesterolaemia; HeFH, heterozygous familial hypercholesterolaemia; HIS, high-intensity statin; HoFH, homozygous familial hypercholesterolaemia; HTN, hypertension; LDL-C, low-density lipoprotein-cholesterol; Lp(a), lipoprotein(a); NA, not applicable; Q2W, administered every 2 weeks; Q4W, administered every 4 weeks; RCT, randomised controlled trial; TG, triglycerides.